Things went very well today for Abeona Therapeutics because it had received FDA approval of the first and only autologous cell-based gene therapy for the treatment of adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB) as a one-time application. This is a devastating rare genetic skin disorder that is marked by fragile skin. These patients suffer from open wounds, blistering, and scarring leading to a loss in quality of life.

The reason why this occurs is because such patients have mutations of both copies of the COL7A1 gene, and this leads to the fragile skin I have described above. The problem is that the number of blisters and wounds that appear on someone can vary depending upon how severe their disease is. A problem area with the skin can be observed in 30% of those with less severe disease, whereas in worst-case scenarios it can cover up to 80% of the body’s surface.

What happens with ZEVASKYN is that patients’ own skin cells are modified to express the COL7A1 gene. These sheets are surgically implanted on wounds and areas of disease, whereby the wounds can then heal effectively because of Type VII collagen being in place. This is huge news for patients that currently have no cure.

For starters, ZEVASKYN can act as a long-term cell-based gene therapy treatment option with a single application. Plus, it is not meant to be used alone, in that it can be provided as an option to be given alongside many approved topical treatments as well. FDA approval was given on positive results from the phase 3 VITAL study, which compared the use of ZEVASKYN to matched control wounds in terms of two co-primary endpoints.

The two efficacy endpoints were to see a statistically significant healing of 50% or more from baseline in large chronic RDEB wounds present and pain reduction from baseline using a scale known as Wong-Baker FACES. Both of these endpoints between therapy and control were evaluated over a 6-month period. With respect to wound healing, it was noted that 81% of wounds achieved this 50% or more healing benchmark compared to only 16% of wounds in matched control.

The therapy was shown to be safe and tolerable; thus I don’t see a problem with this hindering the company’s ability to generate revenue. The most common adverse events (AEs) were itch and pain but were only observed in fewer than 5% of patients. That’s not to say that there won’t be something that may not allow the company to do well when sales start to kick off in Q3 of 2025.

The problem is that the price of this gene therapy could end up being around $3.1 million. On the flip side, it is only going to be needed as a one-time gene therapy cell-based option for these RDEB patients. Plus, it goes back to what I said above in that it could act as a complementary therapy to other approved drugs such as Krystal Biotech’s VYJUVEK.

On a separate note, the FDA approval achieved gave Abeona the ability to receive a Rare Disease Priority Review Voucher (PRV). This is given to companies that develop a treatment for rare disorders or ones that are considered to be an unmet medical need. In its release, the company noted that it plans to monetize it.

This is huge news because these things can be sold for an enormous amount of cash. As a matter of fact, these can be sold for about $100 million or more. The purpose of companies buying these up is because they can cut the review time of a potential approved drug by six months or so. It remains to be seen if Abeona can capitalize on this PRV, but it needs to get its commercialization efforts underway to start generating revenues for ZEVASKYN.